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Yahoo
12-05-2025
- Health
- Yahoo
Chronic Pruritus Market is Anticipated to Expand at a CAGR of 8.6% During the Study Period (2020-2034)
The chronic pruritus market is expected to grow across the 7MM, driven by the introduction of innovative therapies like Dupilumab, Linerixibat, and Volixibat, among others. Furthermore, the rising prevalence of chronic pruritus, driven by factors such as an aging population, increased exposure to environmental irritants, a higher occurrence of chronic inflammatory and autoimmune conditions, and improved diagnostic awareness among healthcare professionals, will further bolster the chronic pruritus market growth. LAS VEGAS, May 12, 2025 /PRNewswire/ -- DelveInsight's Chronic Pruritus Market Insights report includes a comprehensive understanding of current treatment practices, chronic pruritus emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Chronic Pruritus Market Report According to DelveInsight's analysis, the market size for chronic pruritus was found to be USD 3.5 billion in the 7MM in 2024. Estimates suggest that dupilumab is expected to generate approximately USD 700 million in the 7MM by 2034. According to DelveInsight's estimates, there were approximately 98 million total prevalent cases of chronic pruritus in the 7MM in 2024. Among these, around 22 million were diagnosed cases. Leading chronic pruritus companies developing emerging therapies, such as Sanofi, Regeneron, GlaxoSmithKline, Galderma, Chugai Pharmaceutical, Mirum Pharmaceuticals, and others, are developing novel chronic pruritus drugs that can be available in the chronic pruritus market in the coming years. The promising chronic pruritus therapies in the pipeline include DUPIXENT (dupilumab), Linerixibat (GSK2330672), NEMLUVIO/MITCHGA (nemolizumab), Volixibat, and others. In November 2024, GSK reported positive results from the global Phase III GLISTEN trial of linerixibat in adults with cholestatic pruritus in PBC at The Liver Meeting, hosted by the American Association for the Study of Liver Diseases (AASLD). Moreover, the company anticipates regulatory decisions in the U.S. in 2025, and in Europe and Japan in 2026. In November 2024, Mirum Pharmaceuticals presented interim results from the Phase IIb VINTAGE trial of volixibat for cholestatic pruritus in PBC at the AASLD. In September 2024, the Phase III LIBERTY-CPUO-CHIC trial evaluating DUPIXENT in adults with uncontrolled, severe chronic pruritus of unknown origin did not meet statistical significance for its primary itch responder endpoint, though it showed favorable numerical trends. The study achieved nominally significant improvements across all other itch-related endpoints. The Phase III program includes Study A (LIBERTY-CPUO-CHIC) and the planned pivotal Study B. Discover which therapies are expected to grab the major chronic pruritus market share @ Chronic Pruritus Market Report Chronic Pruritus Overview Chronic pruritus, defined as itching that persists for more than six weeks, can severely affect a person's quality of life and is linked to a variety of skin-related and systemic conditions. Its underlying mechanisms involve complex interactions between the immune system and the nervous system, with elements such as cytokines and neuropeptides playing key roles in maintaining the itch. This complexity makes diagnosis challenging, necessitating detailed skin evaluations to distinguish between primary skin issues and secondary changes. Given its diverse origins, effective management requires individualized treatment plans that address the root cause. Diagnosing chronic pruritus demands a structured and thorough approach due to its broad range of possible etiologies. The diagnostic process begins with an in-depth medical history and physical examination to detect any skin abnormalities or signs of systemic illness. Routine lab tests are typically ordered to evaluate the function of the liver, kidneys, and thyroid, along with checks for iron levels and inflammatory markers. Additional procedures, such as skin scrapings or biopsies, may be necessary to exclude infections or underlying malignancies. Chronic Pruritus Epidemiology Segmentation The chronic pruritus epidemiology section provides insights into the historical and current chronic pruritus patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The chronic pruritus market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Chronic Pruritus Total Diagnosed Prevalent Cases of Chronic Pruritus Gender-specific Diagnosed Prevalent Cases of Chronic Pruritus Age-specific Diagnosed Prevalent Cases of Chronic Pruritus Severity-specific Diagnosed Prevalent Cases of Chronic Pruritus Etiology-specific Diagnosed Prevalent Cases of Chronic Pruritus Chronic Pruritus Treatment Market Chronic itch demands a comprehensive treatment plan tailored to its specific cause and biological mechanisms. Successfully managing this condition depends on pinpointing the origin—whether it stems from a skin disorder, an internal disease, a nerve-related issue, or psychological factors—before applying targeted therapies to relieve discomfort. As scientific understanding grows, care is evolving from broad symptom control toward precision medicine, using new drugs that specifically interrupt the pathways driving itch. When itch accompanies skin diseases such as atopic dermatitis or psoriasis, topical corticosteroids remain the mainstay for reducing inflammation and easing itching. By dampening the immune response, these medications lessen redness, swelling, and discomfort. In areas where steroids aren't suitable or for patients who can't use them, calcineurin inhibitors offer a steroid-free alternative. These drugs block calcineurin—a key protein in T-cell activation—thereby curbing inflammation without the typical steroid side effects. In more serious cases of atopic dermatitis, patients often receive systemic biologic treatment such as dupilumab. Dupilumab is a monoclonal antibody that inhibits the IL-4 and IL-13 signaling pathways—major contributors to Type 2 inflammation—leading to symptom relief and strengthened skin barrier function. For chronic kidney disease–associated pruritus (CKD-aP), therapeutic choices now include difelikefalin, a selective kappa-opioid receptor agonist. Given by intravenous infusion, difelikefalin acts on peripheral sensory neurons and immune cells to reduce itching, while its minimal penetration into the central nervous system helps avoid typical opioid-related central side effects. The current treatment landscape for chronic pruritus therapeutics market is relatively sparse, with only a handful of approved options: KORSUVA/KAPRUVIA (Kappa opioid receptor agonist), LIVMARLI (IBAT inhibitor), and BYLVAY/KAYFANDA (IBAT inhibitor). Notably, in March 2025, Japan approved LIVMARLI as the first and so far only therapy for cholestatic itching in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). To know more about chronic pruritus treatment guidelines, visit @ Chronic Pruritus Management Chronic Pruritus Pipeline Therapies and Key Companies DUPIXENT (dupilumab): Sanofi/Regeneron Linerixibat (GSK2330672): GlaxoSmithKline NEMLUVIO/MITCHGA (nemolizumab): Galderma/Chugai Pharmaceutical Volixibat: Mirum Pharmaceuticals Discover more about chronic pruritus drugs in development @ Chronic Pruritus Clinical Trials Chronic Pruritus Market Dynamics The chronic pruritus market dynamics are expected to change in the coming years. A multidisciplinary collaboration among dermatologists, neurologists, allergists, and psychologists enhances diagnostic accuracy and enables personalized treatment, ensuring a comprehensive approach to managing chronic pruritus, which can be effectively treated with peripheral Kappa-Opioid Receptor (KOR) agonists that reduce itch while minimizing central side effects and improving tolerability; moreover, the limited industry focus on chronic pruritus—compared to broader pruritus—highlights an opportunity for innovation in specialized therapies, while a greater emphasis on quality-of-life metrics supports the development of care models that address both the psychosocial burden and symptom control, ultimately improving overall patient outcomes. Furthermore, potential therapies are being investigated for the treatment of chronic pruritus, and it is safe to predict that the treatment space will significantly impact the chronic pruritus market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the chronic pruritus market in the 7MM. However, several factors may impede the growth of the chronic pruritus market. Chronic pruritus of unknown origin remains challenging to manage due to the absence of reliable biomarkers, the failure to effectively target neural sensitization, the dominance of traditional treatments like antihistamines and corticosteroids, and the diverse underlying etiologies—including systemic diseases—all of which hinder precise diagnosis, delay effective treatment, and create barriers to the adoption of newer, more innovative therapies. Moreover, chronic pruritus treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the chronic pruritus market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the chronic pruritus market growth. Chronic Pruritus Market Report Metrics Details Study Period 2020–2034 Coverage 7MM [The United States, the EU4 (Germany, France, Italy, and Spain) and The United Kingdom, and Japan]. Chronic Pruritus Market CAGR 8.6 % Chronic Pruritus Market Size in 2023 USD 3.5 Billion Key Chronic Pruritus Companies Sanofi, Regeneron, GlaxoSmithKline, Galderma, Chugai Pharmaceutical, Mirum Pharmaceuticals, and others Key Pipeline Chronic Pruritus Therapies DUPIXENT (dupilumab), Linerixibat (GSK2330672), NEMLUVIO/MITCHGA (nemolizumab), Volixibat, and others Scope of the Chronic Pruritus Market Report Therapeutic Assessment: Chronic Pruritus current marketed and emerging therapies Chronic Pruritus Market Dynamics: Key Market Forecast Assumptions of Emerging Chronic Pruritus Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Chronic Pruritus Market Access and Reimbursement Download the report to understand which factors are driving chronic pruritus market trends @ Chronic Pruritus Market Trends Table of Contents 1 Key Insights 2 Report Introduction 3 Chronic Pruritus Market Overview at a Glance 3.1 Market Share (%) Distribution of Chronic Pruritus by Therapies in the 7MM in 2020 3.2 Market Share (%) Distribution of Chronic Pruritus by Therapies in the 7MM in 2034 4 Executive Summary 5 Key Events 6 Disease Background and Overview 6.1 Introduction 6.2 Etiology 6.3 Pathophysiology 6.4 Diagnosis 6.4.1 Diagnostic Algorithm 6.4.2 Diagnostic Guidelines 6.5 Treatment and Management 6.5.1 Treatment Algorithm 6.5.2 Treatment Guidelines 7 Methodology 8 Epidemiology and Patient Population 8.1 Key Findings 8.2 Assumptions and Rationale: The 7MM 8.2.1 Total Prevalent Cases of Chronic Pruritus 8.2.2 Total Diagnosed Prevalent Cases of Chronic Pruritus 8.2.3 Gender-specific Cases of Chronic Pruritus 8.2.4 Age-specific Cases of Chronic Pruritus 8.2.5 Severity-specific Cases of Chronic Pruritus 8.2.6 Etiology-specific Cases of Chronic Pruritus 8.3 Total Prevalent Cases of Chronic Pruritus in the 7MM 8.4 Total Diagnosed Prevalent Cases of Chronic Pruritus in the 7MM 8.5 The US 8.5.1 Total Prevalent Cases of Chronic Pruritus in the US 8.5.2 Total Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.5.3 Gender-specific Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.5.4 Age-specific Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.5.5 Severity-specific Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.5.6 Etiology-specific Diagnosed Prevalent Cases of Chronic Pruritus in the US 8.6 EU4 and the UK 8.6.1 Total Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.2 Total Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.3 Gender-specific Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.4 Age-specific Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.5 Severity-specific Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.6.6 Etiology-specific Diagnosed Prevalent Cases of Chronic Pruritus in EU4 and the UK 8.7 Japan 8.7.1 Total Diagnosed Prevalent Cases of Chronic Pruritus in Japan 8.7.2 Gender-specific Diagnosed Prevalent Cases of Chronic Pruritus in Japan 8.7.3 Age-specific Diagnosed Prevalent Cases of Chronic Pruritus in Japan 8.7.4 Severity-specific Diagnosed Prevalent Cases of Chronic Pruritus in Japan 8.7.5 Etiology-specific Diagnosed Prevalent Cases of Chronic Pruritus in Japan 9 Patient Journey 10 Marketed Drugs 10.1 Key Cross Competition 10.2 KORSUVA/KAPRUVIA (difelikefalin): Cara Therapeutics/CSL/ Vifor/Maruishi 10.2.1 Product Description 10.2.2 Regulatory Milestones 10.2.3 Other Developmental Activities 10.2.4 Clinical Trials Information 10.2.5 Safety and Efficacy 10.3 LIVMARLI (maralixibat): Mirum Pharmaceuticals/Takeda 10.3.1 Product Description 10.3.2 Regulatory Milestones 10.3.3 Other Developmental Activities 10.3.4 Clinical Trials Information 10.3.5 Safety and Efficacy 10.4 BYLVAY/KAYFANDA (odevixibat): Ipsen/Jadeite Medicines 10.4.1 Product Description 10.4.2 Regulatory Milestones 10.4.3 Other Developmental Activities 10.4.4 Clinical Trials Information 10.4.5 Safety and Efficacy List to be continued in the final report. 11 Emerging Drugs 11.1 Key Cross Competition 11.2 DUPIXENT (dupilumab): Sanofi/Regeneron 11.2.1 Drug Description 11.2.2 Other Developmental Activities 11.2.3 Clinical Trials Information 11.2.4 Analysts' View 11.3 Linerixibat (GSK2330672): GlaxoSmithKline 11.3.1 Drug Description 11.3.2 Other Developmental Activities 11.3.3 Clinical Trials Information 11.3.4 Safety and Efficacy 11.3.5 Analysts' View 11.4 NEMLUVIO/MITCHGA (nemolizumab): Galderma/Chugai Pharmaceutical 11.4.1 Drug Description 11.4.2 Other Developmental Activities 11.4.3 Clinical Trials Information 11.4.4 Safety and Efficacy 11.4.5 Analysts' View 11.5 Volixibat: Mirum Pharmaceuticals 11.5.1 Drug Description 11.5.2 Other Developmental Activities 11.5.3 Clinical Trials Information 11.5.4 Safety and Efficacy 11.5.5 Analysts' View List to be continued in the final report. 12 Chronic Pruritus – 7MM Market Analysis 12.1 Key Findings 12.2 Key Market Forecast Assumptions 12.2.1 Cost Assumptions and Rebates 12.2.2 Pricing Trends 12.2.3 Analogue Assessment 12.2.4 Launch Year and Therapy Uptake 12.3 Market Outlook 12.4 Attribute Analysis 12.5 Total Market Size of Chronic Pruritus in the 7MM 12.6 Market Size of Chronic Pruritus by Therapies in the 7MM 12.7 The US Market Size 12.7.1 Total Market Size of Chronic Pruritus in the US 12.7.2 Market Size of Chronic Pruritus by Therapies in the US 12.8 EU4 and the UK Market Size 12.8.1 Total Market Size of Chronic Pruritus in EU4 and the UK 12.8.2 Market Size of Chronic Pruritus by Therapies in EU4 and the UK 12.9 Japan Market Size 12.9.1 Total Market Size of Chronic Pruritus in Japan 12.9.2 Market Size of Chronic Pruritus by Therapies in Japan 13 KOL Views 14 Unmet Needs 15 SWOT Analysis 16 Market Access and Reimbursement 16.1 The United States 16.1.1 CMS 16.2 EU4 and the UK 16.2.1 Germany 16.2.2 France 16.2.3 Italy 16.2.4 Spain 16.2.5 The United Kingdom 16.3 Japan 16.3.1 MHLW 17 Appendix 17.1 Acronyms and Abbreviations 17.2 Bibliography 17.3 Report Methodology 18 DelveInsight Capabilities 19 Disclaimer 20 About DelveInsight Related Reports Psoriasis Market Psoriasis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key psoriasis companies including AnaptysBio, Nimbus Lakshmi, Takeda, MoonLake Immunotherapeutics, Bristol-Myers Squibb, Amgen, Dermavant Sciences, Inc., Janssen Research & Development, LLC, Sun Pharmaceutical Industries Limited, UCB Pharma, ACELYRIN Inc., Novartis, among others. 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Atopic Dermatitis Pipeline Atopic Dermatitis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key atopic dermatitis companies, including Kymab, BiomX, LEO Pharma, GlaxoSmithKline, Arjil Pharmaceuticals, SCM Lifescience, Sun Pharmaceutical Industries Limited, Brickell Biotech Inc, Dermira, AstraZeneca, Kyowa Kirin, UCB Biopharma, Arcutis Biotherapeutics, and others. Kymab, BiomX, LEO Pharma, GlaxoSmithKline, Arjil Pharmaceuticals, SCM Lifescience, Sun Pharmaceutical Industries Limited, Brickell Biotech Inc, AstraZeneca, Kyowa Kirin, UCB Biopharma, Arcutis Biotherapeutics, Vanda Pharmaceuticals, Kyowa Kirin, Sanofi, KeyMed Biosciences, Asana BioSciences, Bristol-Myers Squibb, RAPT Therapeutics, Allakos, Novartis, BioMimetix, Shanghai Hengrui Pharmaceutical Co, Connect Biopharma, Pfizer, Evommune, Inc., Fresh Tracks Therapeutics, Biosion, Chia Tai Tianqing Pharmaceutical, Reistone Biopharma Company Limited, JW Pharmaceutical, Oneness Biotech, Alphyn Biologics, selectION, UNION Therapeutics, Ichnos Scien, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur info@ + Logo: View original content: SOURCE DelveInsight Business Research, LLP Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
07-05-2025
- Business
- Business Wire
Mirum Pharmaceuticals Reports First Quarter 2025 Financial Results and Provides Business Update
- - First quarter 2025 total revenue of $111.6 million; 2025 guidance increased to $435 to $450 million - VISTAS study in PSC expected to complete enrollment in third quarter of 2025; topline data expected in second quarter of 2026 - LIVMARLI oral tablet formulation FDA approved - Conference call to provide business updates today, May 7 at 1:30 p.m. PT/4:30 p.m. ET FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the first quarter 2025 and provided a business update. 'It's been a strong start to the year with commercial growth and multiple milestones achieved across our pipeline,' said Chris Peetz, chief executive officer of Mirum. 'We're pleased with the FDA's approval of LIMARLI's tablet formulation, which provides more options for the ALGS and PFIC community in the U.S. in the form of a convenient single tablet dose. Our VISTAS study of volixibat in PSC is enrolling well and we expect to complete enrollment in the third quarter of this year. We're excited to continue our strong execution across our commercial medicines and pipeline throughout the year.' Commercial: Raising full year revenue guidance to $435 to $450 million First quarter 2025 global net product sales of $111.6 million. First quarter 2025 LIVMARLI net product sales were $73.2 million representing 71% growth over first quarter 2024 net product sales. Bile Acid Medicines net product sales were $38.4 million representing 47% growth over first quarter 2024 net product sales. LIVMARLI oral tablet formulation approved. LIVMARLI approved in Japan for ALGS and PFIC. CTEXLI (chenodiol) approved in the US for cerebrotendinous xanthomatosis. Regulatory and Pipeline: Clinical milestones on track Volixibat VISTAS study in primary sclerosing cholangitis (PSC) expected to complete enrollment in third quarter of 2025; topline data expected in the second quarter of 2026. Oral presentation of 28-week interim data from volixibat in PBC (VANTAGE study) at EASL on Friday May 9th at 9:30am CEST. Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in 2026. LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions expected to complete enrollment in 2026. Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in 2025. Corporate and Financial: Strong balance sheet and financial independence Total revenue for the quarter ended March 31, 2025, was $111.6 million compared to $69.2 million for the quarter ended March 31, 2024. Total operating expenses were $126.8 million for the quarter ended March 31, 2025 compared to $95.7 million for the quarter ended March 31, 2024. Total operating expenses for the quarter ended March 31, 2025 included $21.9 million of non-cash stock-based compensation, intangible amortization, and other non-cash expenses compared to $17.1 million for the quarter ended March 31, 2024. As of March 31, 2025, Mirum had unrestricted cash, cash equivalents and investments of $298.6 million compared to $292.8 million as of December 31, 2024. Business Update Conference Call Mirum will host a conference call today, May 7 th at 1:30 p.m. PT/4:30 p.m. ET, to provide business updates. Join the call using the following details: Conference Call Details: U.S./Toll-Free: +1 833 470 1428 International: +1 404 975 4839 Passcode: 549600 You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI has received orphan designation for ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. EU SmPC Canadian Product Monograph About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About CHOLBAM® (cholic acid) capsules The FDA approved CHOLBAM (cholic acid) capsules in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy. CHOLBAM® (cholic acid) Indication CHOLBAM is a bile acid indicated for Treatment of bile acid synthesis disorders due to single enzyme defects. Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption. LIMITATIONS OF USE The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis. ADVERSE REACTIONS The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. Please see full Prescribing Information for additional Important Safety Information. About CTEXLI™ (chenodiol) tablets CTEXLI™ (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries. IMPORTANT SAFETY INFORMATION CTEXLI can cause side effects, including: Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching. Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection. Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines. US Prescribing Information About Mirum Pharmaceuticals Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, significant increase or continued commercial results for our approved medicines, including continued financial growth, the potential achievement of our yearly financial guidance, continued strong execution across our approved medicines and pipeline, the results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for our product candidates in any indication or any specific territory. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as 'expected,' 'will,' 'could,' 'would,' 'guidance,' 'potential,' 'continue' and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum's Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Mirum Pharmaceuticals, Inc. Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited) March 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 211,822 $ 222,503 Short-term investments 65,852 57,812 Accounts receivable 95,852 78,286 Inventory 22,418 22,403 Prepaid expenses and other current assets 14,874 11,784 Total current assets 410,818 392,788 Restricted cash 425 425 Long-term investments 20,912 12,526 Intangible assets, net 243,845 249,819 Other noncurrent assets 14,245 15,196 Total assets $ 690,245 $ 670,754 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 11,639 $ 14,618 Accrued expenses and other current liabilities 115,938 111,933 Total current liabilities 127,577 126,551 Operating lease liabilities, noncurrent 7,003 7,972 Convertible notes payable, net, noncurrent 308,509 308,082 Other liabilities 13,900 2,509 Total liabilities 456,989 445,114 Commitments and contingencies Stockholders' equity: Preferred stock — — Common stock 5 5 Additional paid-in capital 892,367 870,189 Accumulated deficit (658,858 ) (644,181 ) Accumulated other comprehensive loss (258 ) (373 ) Total stockholders' equity 233,256 225,640 Total liabilities and stockholders' equity $ 690,245 $ 670,754 Expand Contacts Investor Contact: Andrew McKibben ir@ Media Contact: Erin Murphy media@ Industry: Biotechnology Pharmaceutical Health Mirum Pharmaceuticals to Announce First Quarter 2025 Financial Results and Host Conference Call on May 7, 2025 FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals to Announce First Quarter 2025 Financial Results and Host Conference Call on May 7, 2025... Mirum Pharmaceuticals to Present Data at Upcoming Medical Congresses
